FDA Releases Guidelines to Push Industry Development of New Therapies
The U.S. Food and Drug Administration (FDA) has released a guidance document to assist and encourage industry in developing new treatment options for the ... Read more
Updates
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Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more -
KB103 Continues to Show Promise in Treating DEB, Receives RMAT Designation from FDA
KB103, Krystal Biotech‘s investigational topical gene therapy for treating dystrophic epidermolysis bullosa (DEB), continues to show promise in the treatment of skin wounds and ... Read more -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the ... Read more
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Vital Signs Monitor May Lower Risk of Skin Injury in NICU Babies and EB Patients
A skin-like device that measures heart rate, breathing, blood oxygen, blood pressure, and body ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
EB-101, Potential Cell Therapy for RDEB, Seen to Lead to Sustained Wound Healing in Trial
EB-101, a cell therapy candidate from Abeona Therapeutics, induced sustained wound healing lasting for three ... -
RDEB Gene Therapy Candidate FCX-007 Given FDA Regenerative Medicine Advanced Therapy Status
Fibrocell Science’s investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa ... -
First Patient Dosed in Phase 2 Trial Testing RGN-137 for Epidermolysis Bulllosa
The first patient with epidermolysis bullosa has received treatment with RegeneRx Biopharmaceuticals‘ investigational therapy RGN-137 ...
Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests
News
A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more